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Getting Ahead of the Curve

The case for a clinical trials platform for stem cell transplantation

A stem cell transplant is a complex treatment and patients can suffer a number of complications, including infection, relapsed disease and secondary cancers. Of those who receive stem cells from an unrelated donor, a third of patients will not survive their first post-transplant year, and fewer than half will live beyond their fifth year.

As such, there is an urgent need to improve patient outcomes by facilitating more clinical trials.

'We have to find a way of getting ahead of the curve in anticipation of the potentially transformative technologies that are on the horizon, and in some cases already with us.'Sir Hugh Taylor, Chairman, Accelerated Access Review, October 2015

Clinical trials compare current treatments with new innovative options and form a base of evidence in support of new treatments. Trials are hugely important, as they allow doctors and patients to know whether new treatments are safe, whether they have any side effects and whether they are more effective than the standard treatments available.

Currently, fewer than 1 in 20 patients requiring a transplant are entered into prospective clinical trials, compared to 1 in 5 cancer patients. Researchers find it difficult to recruit sufficient numbers of patients to trials, due to a complex regulatory environment and a lack of capacity within individual transplant centres.

Establishing a ‘clinical trials platform’ for stem cell transplantation would respond to this challenge by providing the necessary infrastructure for transplant centres to work together to set up studies, recruit patients and share data.

THE CASE FOR A CLINICAL TRIALS PLATFORM

1. Action is needed to improve patient outcomes. Stem cell transplantation is one of the most effective treatments available for blood cancer, but fewer than half of patients receiving stem cells from an unrelated donor will survive beyond their fifth year post-transplant.

2. Not enough medical research is taking place. Clinical trials are vital in forming the evidence base for innovative new treatments, which can improve patient outcomes. However, fewer than 1 in 20 stem cell transplant patients take part in such studies, compared to 1 in 5 cancer patients.

3. There is a clear consensus on the need for a clinical trials platform. Organisations including Anthony Nolan, Bloodwise, the British Society of Bone Marrow Transplantation, NHS Blood and Transplant and the Cell Therapy Catapult all agree that a new clinical trials platform for stem cell transplantation and immunotherapy should be established. The National Institute of Health Research (NIHR) is also supportive of the initiative.

4. A clinical trials platform would help overcome obstacles to research. A platform would provide the means for transplant centres to work together to set up studies, recruit patients, share data and ultimately deliver innovative treatments to patients more quickly.

6. A clinical trials platform would complement existing research infrastructure within the NHS. A clinical trials platform for stem cell transplantation would complement and build upon the NIHR Clinical Research Network. The NIHR will provide support in proportion to the level of trial activity.

7. A clinical trials platform would help to deliver the Government’s agenda for life sciences. A clinical trials platform will support the Accelerated Access Review’s aim to make the UK a ‘go to’ place for firms developing promising new treatments such as cellular immunotherapies, driving growth in the UK’s life sciences sector.

'Unlocking the power of the NHS to be a ‘test-bed’ for new medical innovations could dramatically reduce the time it takes for patients to gain access to new treatments.'George Freeman MP, Minister for Life Sciences, November 2014.